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Researchers have been working for decades to bring gene therapy to the clinic, yet very few patients have received any effective gene-therapy treatments. But that doesn't mean gene therapy is an impossible dream. Even though gene therapy has been slow to reach patients, its future is very encouraging.


Human gene therapy has made substantial progress since the initiation of the first clinical trials 20 years ago. Here, we summarized important applications of gene transfer protocols in the treatment of various human diseases using different viral vectors. Recent successful trials on the treatment ...


Gene therapy ‘cures’ boy of blood disease that affects millions. So far, gene therapy has only treated rare disorders. Now, for the first time, it has been used to treat a boy with sickle cell ...


Gene therapy has been used to treat severe combined immunodeficiency (SCID), a fatal genetic disease whose sufferers have a profoundly weakened immune system. The type of SCID treated is called SCID-X1, because it is caused by mutations in a gene on the X chromosome.


In the medicine field gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease. The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989.


Unlike the gene therapy trial with Ashanthi, who continued to receive enzyme therapy during her stem cell therapy, this meant researchers were able to assess the exact effectiveness of the gene therapy on its own. This observation also suggested that enzyme therapy may have contributed to the lack of success in gene therapy trials.


Since the advent of gene therapy for genetic diseases, a. General practitioners have been more successful in curing some inherited diseases b. Physicians have disregarded symptom-based treatment in favor of biotechnology c. Health Management Organizations have funded most of the experimental treatments and genetic research projects d.


Gene therapy attempts to treat genetic diseases at the molecular level by correcting what is wrong with defective genes. Clinical research into gene therapy’s safety and effectiveness has just begun. No one knows if gene therapy will work, or for what diseases.


Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often ...


BH 4 therapy has been successful, however only a minor proportion of PKU patients benefit from this treatment. Contrasting data has been generated from studies using LNAA as an alternative to dietary therapy and it has been only recommended for adult PKU patients who do not adhere to the diet.